BioMarin gets go-ahead to start gene therapy clinical trials

15 Jan 2020

BMN 307 represents a potential third PKU treatment option from BioMarin and its second gene therapy clinical program.

BioMarin Pharmaceutical, a pioneer in treatments for the rare disease Phenylketonuria (PKU) and in gene therapy clinical research, has announced that both the FDA and the Medicines and Healthcare Products Regulatory Agency (MHRA) have granted the company Investigational New Drug (IND) status and approved its Clinical Trial Application (CTA), respectively, for its investigational gene therapy candidate BMN 307.

BioMarin gets go-ahead to start gene therapy clinical trials

BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU. BMN 307 will be evaluated to determine whether a single dose of treatment can restore natural Phe metabolism, normalize plasma Phe levels, and enable a normal diet in patients with PKU.

The company expects to start dosing patients in PHEARLESS, a Phase I/II study, in the first quarter of 2020 with product made at commercial scale from its award-winning gene therapy manufacturing facility. The company is actively preparing regulatory submissions to open additional clinical sites in other countries. BMN 307 represents a potential third PKU treatment option from BioMarin and its second gene therapy clinical program. Both the FDA and European Medicines Agency (EMA) have granted BMN 307 Orphan Status.

BMN 307 follows BioMarin's first investigational gene therapy clinical program: valoctocogene roxaparvovec to treat severe hemophilia A, currently in Phase III. Based on data from an interim analysis of the ongoing Phase III study, the EMA validated a Marketing Authorization Application in the EU for valoctocogene roxaparvovec, and the review process has begun. The company also has submitted a Biologics License Application to the FDA and anticipates the review to begin in February 2020.

"With BMN 307, we are joining together our expertise in PKU biology and the knowledge we have gained from developing the only two approved therapies for PKU with our understanding of gene therapy clinical development and manufacturing from our valoctocogene roxaparvovec experience," said Hank Fuchs, President, Worldwide Research and Development at BioMarin. "BioMarin has stood with the PKU community for over 15 years and remains dedicated to continuing to increase the body of medical knowledge in this devastating disease."

Read More

Related news

Ajinomoto introduces fast track platform for drug product manufacturing

Ajinomoto introduces fast track platform for drug product manufacturing

30 Jun 2020

AJILITY is designed to speed novel or existing therapeutics from development to manufacturing and into the clinic.

Read more 
Piramal acquires solid oral dosage drug product facility from G&W Laboratories

Piramal acquires solid oral dosage drug product facility from G&W Laboratories

22 Jun 2020

The site can also produce liquids, creams, and ointments, further expanding the CDMO's portfolio.

Read more 
Catalent and ViralClear Pharmaceuticals partner on potential COVID-19 treatment

Catalent and ViralClear Pharmaceuticals partner on potential COVID-19 treatment

22 Jun 2020

The CDMO will work on developing a solution and a solid oral dosage form of ViralClear's merimepodib.

Read more 
Sanofi to build EUR 490 million vaccine production facility in France

Sanofi to build EUR 490 million vaccine production facility in France

16 Jun 2020

The French pharma company will also invest EUR 120 million to create a new research center at Marcy-l'Etoile dedicated to vaccines

Read more 
Vektor Pharma finalizes formula for novel transdermal drug delivery system to treat Parkinson's

Vektor Pharma finalizes formula for novel transdermal drug delivery system to treat Parkinson's

8 Jun 2020

Optimizing the dosage form's efficiency reduces the amount of drug required in each patch.

Read more 
Emergent bags $628 million landmark CDMO deal to produce COVID-19 vaccine candidates

Emergent bags $628 million "landmark" CDMO deal to produce COVID-19 vaccine candidates

2 Jun 2020

The company will provide CDMO capabilities, capacities, and expertise to support the US government’s Warp Speed Program to pave the way for innovators to advance COVID-19 programs.

Read more 
Changes needed to prevent controversial pay-for-delay pharma deals

Changes needed to prevent controversial pay-for-delay pharma deals

26 May 2020

Researchers endorse a switch from the current first-filer system in the US to a system that instead rewards the first successful challenger.

Read more 
SeraNova and Carna Biosciences to develop oral formulation of a new active ingredient

SeraNova and Carna Biosciences to develop oral formulation of a new active ingredient

26 May 2020

SeraNovo enters into a second license agreement with Carna Biosciences for development of oral formulation of Carna’s kinase inhibitor.

Read more 
AbCellera and Lilly seal multi-year antibody discovery collaboration

AbCellera and Lilly seal multi-year antibody discovery collaboration

25 May 2020

AbCellera, will use its antibody drug discovery platform to identify antibodies against up to nine targets.

Read more 
HKBU invents nanostructure that stimulates growth of stem cells for Parkinson's treatment

HKBU invents nanostructure that stimulates growth of stem cells for Parkinson's treatment

25 May 2020

The invention could pave the way for treatments to cure Parkinson's and Alzheimer's disease.

Read more